Background
The combination of a hypomethylating agent with venetoclax (VEN) is now standard of care for many adults with AML. This regimen has demonstrated efficacy in blastic plasmacytoid dendritic cell neoplasm (BPDCN), however, to our knowledge, there are no reports of the all oral-regimen of decitabine cedazuridine (DEC) with VEN in these patients. Here we describe a case of BPDCN in first remission after intensive chemotherapy who was successfully bridged to allo-SCT with oral DEC-VEN.
Case
A 21-year-old female presented with an upper back and breast lesions, for which excisional biopsy 8/1/23 demonstrated BPDCN. PET/CT 8/16/23 showed hypermetabolic lesions in bones, bilateral breasts and axillary lymph nodes. Bone marrow biopsy (BmBx) 8/14/23 demonstrated hypocellular marrow with trilineage hematopoiesis, no increase in blasts, 0.8% aberrant myeloid blasts on flow cytometry. CSF studies including cytology and flow cytometry were negative for BPDCN. She initiated treatment with HyperCVAD+ VEN on 8/23/23. She underwent end of cycle 1 BmBx and PET/CT which demonstrated no evidence of disease. She received an additional 3 cycles of HyperCVAD + VEN consolidation, however was then lost to follow-up. She returned to care 2/2024, at which time restaging indicated ongoing CR. While awaiting donor availability for transplant, she initiated oral DEC x 3 days with VEN x 14 days for 3 cycles which she tolerated well. Pre-transplant BmBx and PET/CT were negative for BPDCN. She proceeded with a 9/10 MMUD allogeneic transplant on 6/8/24. At Day 28, she has 100% engraftment with a negative PET/CT.
Conclusion
To our knowledge, this demonstrates the first case of a patient with BPDCN who was maintained in a CR with an all-oral regimen of DEC with VEN as a bridging modality while awaiting transplant, suggesting it may be a viable option as maintenance or bridging therapy for patients in remission from BPDCN.